NeuBase Therapeutics, Inc. (NASDAQ:NBSE) was founded in 2009 and is headquartered in Pittsburgh, Pennsylvania, USA. It has 9 full-time employees. It is a pre-clinical biopharmaceutical company dedicated to the development of treatments for rare genetic diseases and cancers caused by mutant genes. therapy.
NeuBase Therapeutics, Inc. (NBSE):
NeuBase Therapeutics is developing next-generation gene silencing therapies through its flexible, highly specific synthetic antisense oligonucleotides.
The proprietary NeuBase Peptide Nucleic Acid Antisense Oligonucleotide (PATrOL™) platform enables rapid development of targeted drugs, thereby increasing treatment opportunities for hundreds of millions of people affected by rare genetic diseases, including those that can only be accessed through secondary RNA Structure to treat diseases. By using PATrOL technology, NeuBase aims to solve rare genetic neurological diseases first.
NeuBase Therapeutics’ proprietary peptide-nucleic acid antisense oligonucleotide (PATrOL) platform is dedicated to solving Huntington’s disease (HD), tonic dystrophy and other genetic diseases. Candidate products include:
- The main plan is NT0100, which is a therapeutic plan that supports PATrOL for systemic drug delivery, targeting mutant extensions in HD messenger ribonucleic acid (mRNA);
- NT0200, a therapeutic program with PATrOL function, is used for systemic administration to target the treatment of mutants in mRNA of type 1 myotonic dystrophy disease.